Product | 06.03.2024
This year´s annual meeting of the European Society of Blood and Marrow Transplantation (EBMT) took place in Paris from April 23rd to 26th. The symposium "Optimizing Conditioning Regimens and Advancing Allogeneic Hemopoietic Stem Cell Transplantation Practice", organised by Medscape and supported with an educational grant from medac was – as well as an additional Meet-The-Expert-Session focussed on paediatric patients the following day – extremely well attended and of high interest. The recent approval for paediatric patients with non-malignant diseases has expanded the opportunities for discussions.1
Trecondi® is the only Treosulfan in Europe approved in allogeneic haematopoietic stem cell transplantation (alloHSCT). Treosulfan in combination with Fludarabine is indicated as part of conditioning treatment prior to alloHSCT in adult patients with malignant and non-malignant diseases, and in paediatric patients older than one month with malignant and non-malignant diseases.1
The scientific symposium “Optimizing Conditioning Regimens and Advancing Allogeneic Hemopoietic Stem Cell Transplantation Practice” was moderated by Friedrich Stölzel (Germany) with Matthias Stelljes (Germany), Filippo Milano (USA), and Domenico Russo (Italy) as presenting experts.
In his presentation at the beginning of the symposium, Stölzel provided an overview on the use of alloHSCT in patients with myelodysplastic syndrome (MDS) and acute myeloid leukaemia (AML), their eligibility for alloHSCT and which ones will particularly benefit. With alloHSCT being the only potentially curative therapy for patients with MDS or high-risk AML, new risk stratifications and redefined overlapping myeloid disease entities might lead to higher or earlier alloHSCT rates. Thus, the concept of either myeloablative conditioning (MAC) or reduced intensity conditioning (RIC) has been expanded to include recently developed reduced toxicity conditioning (RTC). The boundaries from RIC to RTC and RTC to MAC are blurry, as are the boundaries of patients’ disease characteristics and risk factors leading to a higher or lesser benefit from a particular conditioning regimen. Stölzel concluded that RIC and RTC strategies might offer the best of both worlds for patients with MDS and AML while current literature and observations merit further study in prospective andomized clinical trials (RCTs).
Stelljes presented alloHSCT as a highly effective treatment option for patients with standard and high-risk AML. A prospective, randomised, open label, multicenter phase 3 trial 2 showed the advantage of Treosulfan-Fludarabine (Treo/Flu) compared to Busulfan-Fludarabine (Bu/Flu) before alloHSCT in older and/or comorbid adult patients with AML or MDS. Patients treated with the Treo/Flu regimen showed a significantly improved event-free survival (EFS) of 59.5% at 3 years compared to 49.7% with Bu/Flu (p=0.0005787), due to a significantly lower non-relapse mortality of 14.2% vs. 21.0% (p=0.0392). Furthermore, real world data using Treosulfan-based conditioning before alloHSCT for older patients with MDS3 including patients with high-risk disease showed a promising survival outcome of 72% at 2 years, further strengthening the use of this regimen in this patient population.
Stelljes concluded that during the last 20 years the transplant outcomes had significantly improved and underlined that the conditioning regimen with Treosulfan and Fludarabine is a new standard option before alloHSCT in older adult and/or comorbid patients with AML and MDS. “A 2-year survival rate of >70% for patients aged 50 to 70 years or younger adult patients with comorbidities clearly supports alloHSCT as a highly efficient post-remission therapy for these patients.” emphasised Stelljes.
Milano presented the Seattle experience on the use of a Treosulfan-based conditioning regimen. In a recent trial using this regimen, cord blood recipients with AML, MDS and ALL had a 57% probability of disease-free survival at 3 years 4. These results led to a new study 5 using Treosulfan-based conditioning for haploidentical transplantation which is currently recruiting patients. Milano concluded that, while novel conditioning regimens provide promising results regarding response and low relapse risk, clinical trials are of continuing importance.
In the last lecture of the symposium Russo discussed therapy options based on two patient cases. The audience in the room had the opportunity to interactively vote on treatment options after considering the challenges in treating elderly patients with alloHSCT, based on disease and patient characteristics. It was clearly shown that any treatment decision in this field is multifactorial, precluding any “one-treatment-fits all” approach.
In the “Meet The Expert” 6 session on Monday afternoon, a selected group of international physicians had the chance to discuss the use of Trecondi® in paediatric patients with the expert Krzysztof Kalwak (Poland). After an introduction to medac’s paediatric clinical trial program, highlighting the excellent results in terms of efficacy and very low treatment-related mortality, an interesting discussion ensued, focusing on further details like quality of life, dosing, and the versatility of Trecondi® as a conditioning agent in a variety of indications.
Since 2019 Trecondi® is the only Treosulfan in European countries approved as part of conditioning therapy in combination with Fludarabine prior to allogeneic haematopoietic stem cell transplantation in adult and paediatric patients older than one month with malignant and non-malignant diseases. Trecondi® is manufactured by medac GmbH subsidiary Oncotec Pharma Production GmbH in Germany. Find out more at www.trecondi.com
